Novel Ex Vivo Gene Therapy Approaches and
Emerging Gene Editing Technologies


Thursday, September 12, 2019
Monona Terrace Community and Convention Center
1 John Nolen Drive
Madison, Wisconsin 53703

 

Symposium Overview|Final Program|Supporters

Developed by the ISCT Immuno-Gene Therapy Committee, this full day symposium offers the unique opportunity to gather key opinion leaders in the field of Gene Therapy to discuss, present and develop position statements to move the field forward.

Aimed at driving thought leadership by providing content and building collaboration, the ISCT Gene Therapy Signature Series will address a variety of novel ex-vivo gene therapy approaches and emerging gene editing technologies from the translational science, manufacturing and regulatory perspectives.

To facilitate in-depth discussion and promote collaboration among all speakers and participants, the Scientific Signature Series is limited to a select number of attendees by invitation only.

To be eligible to participate in the ISCT Gene Therapy Signature Series, applicants must...

    1. Be registered for the ISCT North America 2019 Regional Meeting, September 13-15. CLICK HERE to register today!
    2. Actively contribute to the field of Gene Transduction/Gene Therapy.
    3. Receive a formal invitation to participate in the ISCT 2019 Gene Therapy Signature Series.

Please note there is no  registration fee to participate in the Signature Series event; however, there are limited seats available for a select number of attendees by invitation only.

Program


HSPC Selection, Conditioning and the Synthetic Biology Platform
8:30 AM-10:00 AM

    • Speakers:
      • Hans-Peter Kiem, MD, PhD, Fred Hutchinson Cancer Research Center, USA
        • TARGETING A HIGHLY ENRICHED HEMATOPOIETIC STEM CELL POPULATION FOR GENE THERAPY AND EDITING
      • Anthony Boitano, PhD, Magenta Therapeutics, USA
        • A CD117-AMANITIN ANTIBODY DRUG CONJUGATE (ADC) EFFECTIVELY DEPLETES HUMAN AND NON-HUMAN PRIMATE HEMATOPOIETIC STEM AND PROGENITOR CELLS (HSPCS): TARGETED NON-GENOTOXIC CONDITIONING FOR BONE MARROW TRANSPLANT
      • Philip Lee, PhD, Senti Biosciences, USA
        • MANUFACTURING DEVELOPMENT OF ALLOGENEIC CELL THERAPIES POWERED BY GENE CIRCUITS

 

The CRISPR-Cas Gene-Editing System
10:15 AM-11:45 AM

    • Speakers:
      • Matthew Porteus, MD, PhD, Stanford University, USA
        • PROGRESS AND CHALLENGES IN DEVELOPING A CAS9 BASED HEMATOPOIETIC STEM CELL DRUG
      • Paula Rio, PhD, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Spain
        • NHEJ-MEDIATED GENE EDITING: AN ALTERNATIVE APPROACH TO EFFICIENTLY CORRECT HSPCS FROM PATIENTS WITH FANCONI ANEMIA
      • Robert Ihry, PhD, Novartis Institutes for Biomedical Research, USA
        • ROLE OF P53 IN CRISPR-CAS9 EDITING

 

Advances in Gene Therapy Technologies and Applications
12:45 PM-2:30 PM

    • Speakers:
      • Krishanu Saha, PhD, University of Wisconsin-Madison, USA
        • PRECISE GENE EDITING OF MUTANT ALLELES USING CRISPR-CAS9 RIBONUCLEOPROTEIN COMPLEXES
      • Daniel Bauer, MD, PhD, Boston Children’s Hospital, USA
        • GENE EDITING FOR FETAL HEMOGLOBIN INDUCTION
      • Melissa Bonner, PhD, bluebird bio, USA
        • USE OF LENTIVIRAL VECTORS IN HSCS FOR TREATMENT OF MONOGENIC DISEASE
      • Isabelle Rivière, PhD, Memorial Sloan Kettering Cancer Center, USA
        • ESTABLISHING CGMP MANUFACTURING OF CRISPR/CAS9-EDITED HUMAN CAR T CELLS


Regulatory & Manufacturing Considerations

2:45 PM-4:15 PM

    • Speakers:
      • Sarah Nikiforow, MD, PhD, Dana-Farber Cancer Institute, USA
        • NUANCES OF GENE THERAPY PRODUCT MANUFACTURING, ANALYSIS AND QUALITY TESTING
      • Neehar Bhatia, PhD, Stanford University, USA
        • DEVELOPING A CGMP MANUFACTURING PROCESS FOR CELL AND GENE THERAPIES
      • Peter Marks, MD, PhD, Center for Biologics Evaluation and Research, FDA, USA
        • ADVANCING INDIVIDUALIZED GENE THERAPY

 

Chair, ISCT Gene Therapy Signature Series:
Sandeep Soni, MD, Stanford University, USA

Organized by the ISCT Immuno-Gene Therapy Committee. For more information on committee initiatives and membership CLICK HERE!

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